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ClinicalTrials.gov public records Last synced Oct 23, 2026, 7:01 AM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 913–936 of 8,007 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Genetics/Birth Defects Clear all
Terminated Not applicable Interventional Results available

Trial of RiaSTAP Versus Cryoprecipitate to Lower Operative Transfusions

NCT02540434
View directory record Official record
Conditions
Coagulopathy
Interventions
ROTEM, Cryoprecipitate, RiaSTAP
Device · Other · Drug
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
18 Years to 100 Years
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jan 15, 2019 · Synced Oct 23, 2026, 7:01 AM EDT
Completed Phase 2 Interventional

Idebenone to Treat Friedreich's Ataxia

NCT00229632
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Idebenone
Drug
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
9 Years to 17 Years
Enrollment
51 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2007
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 18, 2019 · Synced Oct 23, 2026, 7:01 AM EDT
Terminated Phase 2 Interventional Results available

A Dose Finding Study to Investigate the Safety and Effectiveness of GSK1070806 in Adult Participants With Moderate to Severe Atopic Dermatitis

NCT05999799
View directory record Official record
Conditions
Dermatitis, Atopic
Interventions
GSK1070806, Placebo
Drug
Lead sponsor
GlaxoSmithKline
Industry
Eligibility
18 Years to 75 Years
Enrollment
161 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
17
States / cities
Phoenix, Arizona • North Little Rock, Arkansas • Canoga Park, California + 13 more
Source: ClinicalTrials.gov public record
Updated Jun 3, 2026 · Synced Oct 23, 2026, 7:01 AM EDT
Completed No phase listed Observational

Impact of Unapproved Drug Initiative on Colchicine Use

NCT02000232
View directory record Official record
Conditions
Gout
Interventions
Not listed
Lead sponsor
Stanford University
Other
Eligibility
18 Years and older
Enrollment
185 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
1
States / cities
Stanford, California
Source: ClinicalTrials.gov public record
Updated Aug 10, 2022 · Synced Oct 23, 2026, 7:01 AM EDT
Completed No phase listed Observational

Understanding Fabry Disease Therapy Choices Through the Eyes of the Patients

NCT04804566
View directory record Official record
Conditions
Fabry Disease
Interventions
This is a non-interventional study
Other
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023
U.S. locations
1
States / cities
Eagan, Minnesota
Source: ClinicalTrials.gov public record
Updated Apr 3, 2024 · Synced Oct 23, 2026, 7:01 AM EDT
Completed Phase 1Phase 2 Interventional

Combination Trial of Tipifarnib and Alpelisib in Adult Recurrent/ Metastatic Head and Neck Squamous Cell Carcinoma (R/M HNSCC)

NCT04997902
View directory record Official record
Conditions
HNSCC
Interventions
Tipifarnib, Alpelisib
Drug
Lead sponsor
Kura Oncology, Inc.
Industry
Eligibility
18 Years and older
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
11
States / cities
Duarte, California • Orlando, Florida • Baltimore, Maryland + 7 more
Source: ClinicalTrials.gov public record
Updated Sep 8, 2026 · Synced Oct 23, 2026, 7:01 AM EDT
Completed No phase listed Observational

The Experience of Caregivers of Children With Down Syndrome

NCT00695695
View directory record Official record
Conditions
Down Syndrome
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
18 Years and older
Enrollment
546 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2014
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 17, 2019 · Synced Oct 23, 2026, 7:01 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Specimen and Data Study for Ovarian Cancer Early Detection and Prevention

NCT00005095
View directory record Official record
Conditions
Cervical Cancer, Endometrial Cancer, Fallopian Tube Cancer, Hereditary Breast/Ovarian Cancer (brca1, brca2), Ovarian Cancer, Sarcoma, Uterine Leiomyomata, Vaginal Cancer, Vulvar Cancer
Interventions
Ca-125, screening questionnaire administration, Surgery
Other · Procedure
Lead sponsor
Northwestern University
Other
Eligibility
18 Years to 80 Years · Female only
Enrollment
6,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2000 – 2040
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Feb 28, 2025 · Synced Oct 23, 2026, 7:01 AM EDT
Terminated Early Phase 1 Interventional

18F-FES PET/CT in Imaging Patients With Desmoid Tumors

NCT02374931
View directory record Official record
Conditions
Deep Fibromatosis/Desmoid Tumor, Familial Adenomatous Polyposis
Interventions
F-18 16 Alpha-Fluoroestradiol, Positron Emission Tomography, Computed Tomography, Laboratory Biomarker Analysis
Drug · Procedure · Other
Lead sponsor
Vanderbilt-Ingram Cancer Center
Other
Eligibility
18 Years and older
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Oct 3, 2019 · Synced Oct 23, 2026, 7:01 AM EDT
Terminated Not applicable Interventional Results available

Evoked Retinal Response

NCT01415453
View directory record Official record
Conditions
Retinitis Pigmentosa
Interventions
Ultrasound Stimulus
Procedure
Lead sponsor
Columbia University
Other
Eligibility
18 Years to 60 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2012
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Apr 5, 2013 · Synced Oct 23, 2026, 7:01 AM EDT
Completed Phase 1 Interventional

A Phase 1 Study of Givosiran (ALN-AS1) in Patients With Acute Intermittent Porphyria (AIP)

NCT02452372
View directory record Official record
Conditions
Acute Intermittent Porphyria
Interventions
givosiran (ALN-AS1), Sterile Normal Saline (0.9% NaCl)
Drug
Lead sponsor
Alnylam Pharmaceuticals
Industry
Eligibility
18 Years to 65 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
4
States / cities
Birmingham, Alabama • San Francisco, California • New York, New York + 1 more
Source: ClinicalTrials.gov public record
Updated Jun 14, 2018 · Synced Oct 23, 2026, 7:01 AM EDT
Terminated Phase 2 Interventional Results available

Evaluation of the Efficacy and Safety of VX-814 in Subjects With the PiZZ Genotype

NCT04167345
View directory record Official record
Conditions
Alpha 1-Antitrypsin Deficiency
Interventions
VX-814, Placebo
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
18 Years to 80 Years
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020
U.S. locations
22
States / cities
Birmingham, Alabama • Sacramento, California • Denver, Colorado + 19 more
Source: ClinicalTrials.gov public record
Updated Feb 2, 2022 · Synced Oct 23, 2026, 7:01 AM EDT
Completed Phase 2 Interventional Results available

Zoledronate or Observation in Maintaining Bone Mineral Density in Patients Who Are Undergoing Surgery to Remove Both Ovaries

NCT00305695
View directory record Official record
Conditions
Hereditary Breast and Ovarian Cancer Syndrome, Osteoporosis, Ovarian Carcinoma
Interventions
Laboratory Biomarker Analysis, Zoledronic Acid
Other · Drug
Lead sponsor
Gynecologic Oncology Group
Network
Eligibility
Female only
Enrollment
160 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2005
U.S. locations
95
States / cities
Scottsdale, Arizona • Tucson, Arizona • Burbank, California + 76 more
Source: ClinicalTrials.gov public record
Updated Mar 24, 2020 · Synced Oct 23, 2026, 7:01 AM EDT
Recruiting Phase 4 Interventional Accepts healthy volunteers

The Impact of Salt Intake on Sodium in the Skin and Inflammatory Skin Disease

NCT07487831
View directory record Official record
Conditions
Atopic Dermatitis (Eczema), Psoriasis
Interventions
Sodium chloride tablets, Placebo Tablets
Drug · Other
Lead sponsor
University of California, San Francisco
Other
Eligibility
18 Years and older
Enrollment
50 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2026 – 2028
U.S. locations
2
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Sep 9, 2026 · Synced Oct 23, 2026, 7:01 AM EDT
Recruiting Phase 1 Interventional

A Study Assessing KB407 for the Treatment of Cystic Fibrosis

NCT05504837
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
KB407 (Nebulization)
Biological
Lead sponsor
Krystal Biotech, Inc.
Industry
Eligibility
18 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
6
States / cities
Gainesville, Florida • Boise, Idaho • Northfield, Illinois + 3 more
Source: ClinicalTrials.gov public record
Updated May 7, 2026 · Synced Oct 23, 2026, 7:01 AM EDT
Active, not recruiting Not applicable Interventional

Home Based Tele-exercise for People With Chronic Neurological Impairments

NCT04564495
View directory record Official record
Conditions
Neurologic Disorder, Stroke, Spinal Cord Injuries, Cerebral Palsy, Hemiplegia, Hemiparesis, Multiple Sclerosis, Parkinson Disease, Trauma, Brain, Transverse Myelitis, Friedreich Ataxia
Interventions
Seated exercise
Behavioral
Lead sponsor
Burke Medical Research Institute
Other
Eligibility
18 Years to 75 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
1
States / cities
White Plains, New York
Source: ClinicalTrials.gov public record
Updated Feb 5, 2025 · Synced Oct 23, 2026, 7:01 AM EDT
Completed Phase 3 Interventional

Preventing Cytomegalovirus (CMV) Organ Damage With Valganciclovir in People With HIV

NCT00006145
View directory record Official record
Conditions
Cytomegalovirus Infections, HIV Infections
Interventions
Valganciclovir
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
13 Years and older
Enrollment
350 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2006
U.S. locations
56
States / cities
Birmingham, Alabama • Los Angeles, California • Palo Alto, California + 33 more
Source: ClinicalTrials.gov public record
Updated Nov 1, 2021 · Synced Oct 23, 2026, 7:01 AM EDT
Recruiting No phase listed Observational

Monitoring Pregnancy and Infant Outcomes Following Tralokinumab Exposure During Pregnancy in the US and Canada - PROTECT

NCT05938478
View directory record Official record
Conditions
Atopic Dermatitis, Eczema
Interventions
Tralokinumab
Drug
Lead sponsor
University of California, San Diego
Other
Eligibility
Female only
Enrollment
900 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2035
U.S. locations
1
States / cities
La Jolla, California
Source: ClinicalTrials.gov public record
Updated Jan 28, 2025 · Synced Oct 23, 2026, 7:01 AM EDT
Completed No phase listed Observational Results available

The Assessment of Copper Parameters in Wilson Disease Participants on Standard of Care Treatment

NCT02763215
View directory record Official record
Conditions
Wilson Disease
Interventions
Standard of Care Medications
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
64 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
5
States / cities
New Haven, Connecticut • Chicago, Illinois • Ann Arbor, Michigan + 2 more
Source: ClinicalTrials.gov public record
Updated Dec 1, 2020 · Synced Oct 23, 2026, 7:01 AM EDT
Completed Phase 2 Interventional Results available

Pharmacodynamic Study of Emixustat Hydrochloride in Subjects With Macular Atrophy Secondary to Stargardt Disease

NCT03033108
View directory record Official record
Conditions
Stargardt Disease, Macular Atrophy
Interventions
Emixustat
Drug
Lead sponsor
Kubota Vision Inc.
Industry
Eligibility
18 Years and older
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated May 19, 2021 · Synced Oct 23, 2026, 7:01 AM EDT
Completed Phase 2 Interventional Results available

Treatment of Sexual Dysfunction in Hypogonadal Men With Epilepsy With Testosterone and Either Anastrozole or Placebo

NCT00179517
View directory record Official record
Conditions
Seizure Disorder, Hypogonadism, Erectile Dysfunction
Interventions
Anastrozole 1mg, Placebo Oral Tablet
Drug
Lead sponsor
Beth Israel Deaconess Medical Center
Other
Eligibility
18 Years to 50 Years · Male only
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2008
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jul 17, 2019 · Synced Oct 23, 2026, 7:01 AM EDT
Completed No phase listed Observational

Primary Hyperoxaluria Mutation Genotyping/Phenotyping

NCT02340689
View directory record Official record
Conditions
Primary Hyperoxaluria
Interventions
Genetic Analysis
Other
Lead sponsor
Mayo Clinic
Other
Eligibility
1 Day and older
Enrollment
1,235 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2018
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Aug 19, 2019 · Synced Oct 23, 2026, 7:01 AM EDT
Recruiting Not applicable Interventional

Cerebellar TMS and Satiety in Prader-Willi Syndrome

NCT05938543
View directory record Official record
Conditions
Prader-Willi Syndrome
Interventions
Repetitive Transcranial Magnetic Stimulation (rTMS)
Device
Lead sponsor
Brigham and Women's Hospital
Other
Eligibility
18 Years to 64 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
1
States / cities
Belmont, Massachusetts
Source: ClinicalTrials.gov public record
Updated Oct 15, 2026 · Synced Oct 23, 2026, 7:01 AM EDT
Completed Phase 2 Interventional

Safety Study of Roflumilast Cream 0.05% in Infants Aged 3 Months to Less Than 2 Years With Atopic Dermatitis

NCT06998056
View directory record Official record
Conditions
Atopic Dermatitis (Eczema)
Interventions
ARQ-151 cream 0.05%
Drug
Lead sponsor
Arcutis Biotherapeutics, Inc.
Industry
Eligibility
3 Months to 2 Years
Enrollment
101 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025
U.S. locations
17
States / cities
Birmingham, Alabama • Bryant, Arkansas • Fountain Valley, California + 14 more
Source: ClinicalTrials.gov public record
Updated Dec 10, 2025 · Synced Oct 23, 2026, 7:01 AM EDT